Duchenne muscular dystrophy patients treated with GlaxoSmithKline’s (GSK_) experimental drug drisapersen have been hospitalized due to kidney toxicity and low platelet counts, according to a Glaxo scientist who spoke at a research meeting in Rome last Sunday. Glaxo has disclosed some safety data from early-stage drisapersen clinical trials previously, mainly related to moderate cases of proteinuria (excess protein in the urine) and decreased thrombocytes, which play a role in blood clotting. However, the significance of the adverse events attributed to drisapersen, also known as GSK-2402968, escalated with Sunday’s disclosure of hospitalized patients. Dr. Rohit Batta, global medical leader in Glaxo’s neuromuscular rare disease unit, said four Duchenne muscular dystrophy (DMD) patients treated with drisapersen required hospitalization due to thrombocytopenia and that “several” patients with “severe proteinuria” also required hospitalization. Batta’s remarks were made during a presentation on Sunday, Feb. 24 at The XI International Conference on Duchenne Muscular Dystrophy. The conference was held in Rome and organized by Duchenne Parent Project Onlus, an Italian DMD advocacy group. Batta provided no other information about the hospitalized drisapersen patients during his presentation. A Glaxo spokesperson contacted Wednesday confirmed Batta’s remarks and added the DMD patients hospitalized were all enrolled in the ongoing phase II and phase III clinical trials of drisapersen. The hospitalizations had occurred “over the last two years” and had been discussed at previous medical meetings.
